Mondin Alessandro, Ceccato Filippo, Scaroni Carla, Barbot Mattia
Department of Medicine-DIMED, University of Padova, Padova, Italy.
Endocrinology Unit, University Hospital of Padova, Padova, Italy.
Endocr Metab Immune Disord Drug Targets. 2025;25(10):824-833. doi: 10.2174/0118715303328077240719055819.
The possibility of sustained disease remission in functioning pituitary adenomas after drug withdrawal is well-known for prolactinomas and it has also been described in a subset of acromegalic patients. On the contrary, medical treatment for Cushing's Disease (CD) is generally considered a life-long measure except for previously radio-treated patients. Sparse evidence of spontaneous remissions in CD has been reported, mainly related to a possible pituitary tumor apoplexy. To the best of our knowledge, none of these cases has included the use of a pituitary targeting agent.
Herein, we have reported the case of a radiotherapy-naïve patient with persistent CD after pituitary surgery who participated in the CSOMG230 trials, presenting sustained remission after Long-acting Release (LAR) pasireotide withdrawal. Under monthly pasireotide LAR 40 mg, the patient achieved urinary hormone control and clinical signs of cortisol excess normalization. After 8 years of treatment, the patient completed the study protocol and had to withdraw the drug as it was no longer available for CD in Italy. Before starting new therapies, we reassessed hormone levels that were surprisingly within normal ranges. At 24 months after the last dose of pasireotide, the patient was still in clinical and biochemical full remission. We have also briefly reviewed previous cases of sustained remission after somatostatin analogues withdrawal in other functioning pituitary adenomas.
Far from the general rule, this case suggests that prolonged treatment with pasireotide LAR might induce a durable CD remission. A dose down-titration/suspension might be considered in patients well-controlled on long-term therapy and with negative pituitary imaging. However, close monitoring is recommended given the high rate of complications in untreated patients.
泌乳素瘤停药后功能性垂体腺瘤持续疾病缓解的可能性是众所周知的，在一部分肢端肥大症患者中也有描述。相反，除了先前接受过放射治疗的患者外，库欣病（CD）的药物治疗通常被认为是一种终身措施。已有关于CD自发缓解的零星证据报道，主要与可能的垂体瘤卒中有关。据我们所知，这些病例均未包括使用垂体靶向药物。
在此，我们报告了一例垂体手术后持续性CD且未接受过放疗的患者，该患者参加了CSOMG230试验，在长效释放（LAR）帕瑞肽停药后出现持续缓解。在每月40mg帕瑞肽LAR治疗下，患者实现了尿激素控制，皮质醇过量的临床体征恢复正常。经过8年治疗，患者完成了研究方案，因该药物在意大利不再用于CD治疗而不得不停药。在开始新的治疗之前，我们重新评估了激素水平，令人惊讶的是其在正常范围内。在最后一剂帕瑞肽后24个月，患者仍处于临床和生化完全缓解状态。我们还简要回顾了其他功能性垂体腺瘤在生长抑素类似物停药后持续缓解的既往病例。
与一般规律不同，该病例提示帕瑞肽LAR的长期治疗可能诱导CD持久缓解。对于长期治疗控制良好且垂体影像学检查阴性的患者，可考虑降低剂量/暂停用药。然而，鉴于未治疗患者并发症发生率高，建议密切监测。
